Parkinson’s Disease

Ceregene has developed CERE- 120 (AAV-neurturin) to restore lost motor function and protect against further losses in Parkinson’s patients, thus restoring significant quality of life that has been lost. Parkinson’s disease is a devastating and progressive neurodegenerative disease caused by the loss of dopamine neurons deep within the brain. Loss of these neurons leads to motor problems such as difficulty in initiating movement, as well as debilitating tremors and postural instability. PD is a life-taking disease. Even in its earliest stages, difficulties in performing otherwise simple tasks make daily living extremely challenging for those afflicted with this disease.

Treatment of Parkinson’s Disease

Though early in the course of Parkinson’s disease, drugs that enhance dopamine function are able to significantly correct the motor problems, over time these drugs universally become ineffective. In time, patients suffer long periods where movement is not possible or where serious debilitating side effects of the dopamine drugs cannot be avoided. Thus, the need to develop more effective treatments for Parkinson’s disease is widely recognized.

Ceregene’s Parkinson’s Disease Treatment Approach

Ceregene’s approach to treating Parkinson’s disease is different. A single gene transfer procedure is all that is needed to provide symptom relief, indefinitely, while also possibly preventing further disease progression. We have identified neurturin as a neurotrophic factor that can maintain the health and restore the viability of dopamine containing neurons. The gene for this growth factor is delivered directly into dopamine rich regions of the brains of patients with Parkinson’s disease using Ceregene’s gene delivery technology. This technology uses a highly modified and harmless version of adeno-associated virus (AAV) type 2 as the gene delivery vector. The product that Ceregene has developed to perform this feat is called CERE-120. Because neurturin can restore lost function as well as prevent further degeneration of dopamine neurons, CERE-120 is expected not only to reduce the serious motor deficits in the advanced stages of PD, but also may stop or even reverse the loss of these critical neurons, restoring and maintaining motor function in these patients following a single gene transfer procedures.

Ceregene has completed Phase I clinical trials in patients with Parkinson’s disease with CERE-120 and currently has an active multi-center, Phase 2 controlled study. Ceregene has also developed similar products for other terrible neurodegenerative diseases, such as Alzheimer’s disease, ALS (Lou Gehrig’s disease) and several ocular diseases that cause blindness.